CZ2020529A3 - Lipidoidy pro transfekci nukleových kyselin a jejich použití - Google Patents

Lipidoidy pro transfekci nukleových kyselin a jejich použití Download PDF

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Publication number
CZ2020529A3
CZ2020529A3 CZ2020529A CZ2020529A CZ2020529A3 CZ 2020529 A3 CZ2020529 A3 CZ 2020529A3 CZ 2020529 A CZ2020529 A CZ 2020529A CZ 2020529 A CZ2020529 A CZ 2020529A CZ 2020529 A3 CZ2020529 A3 CZ 2020529A3
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CZ
Czechia
Prior art keywords
transfection
lipidoid
mrna
mmol
formula
Prior art date
Application number
CZ2020529A
Other languages
Czech (cs)
English (en)
Inventor
Petr CĂ­gler
Cígler Petr Mgr., Ph.D.
Klára GRANTZ ŠAŠKOVÁ
Grantz Šašková Klára Mgr., Ph.D.
Václav Vaněk
Vaněk Václav Mgr., Ph.D.
Zuzana Kružíková
Zuzana Mgr. Kružíková
František Sedlák
František MUDr. Sedlák
Original Assignee
Ústav organické chemie a biochemie AV ČR, v. v. i.
Ústav organické chemie a biochemie AV ČR, v. v. i.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ăšstav organickĂ© chemie a biochemie AV ÄŚR, v. v. i., Ústav organické chemie a biochemie AV ČR, v. v. i. filed Critical Ăšstav organickĂ© chemie a biochemie AV ÄŚR, v. v. i.
Priority to CZ2020529A priority Critical patent/CZ2020529A3/cs
Priority to AU2021348717A priority patent/AU2021348717B2/en
Priority to CN202180008677.8A priority patent/CN114945556B/zh
Priority to BR112022024346A priority patent/BR112022024346A2/pt
Priority to JP2022530325A priority patent/JP7260717B2/ja
Priority to IL291097A priority patent/IL291097B2/en
Priority to CA3154983A priority patent/CA3154983C/en
Priority to KR1020227011339A priority patent/KR20220057605A/ko
Priority to PCT/CZ2021/050079 priority patent/WO2022063350A1/en
Priority to EP21762590.4A priority patent/EP4031522A1/en
Priority to MX2022013522A priority patent/MX2022013522A/es
Priority to US18/015,254 priority patent/US20230265049A1/en
Publication of CZ2020529A3 publication Critical patent/CZ2020529A3/cs
Priority to ZA2022/03990A priority patent/ZA202203990B/en

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/57—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings
    • C07C233/62—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/24—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atom of at least one of the carboxamide groups bound to a carbon atom of a ring other than a six-membered aromatic ring of the carbon skeleton
    • C07C237/26—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atom of at least one of the carboxamide groups bound to a carbon atom of a ring other than a six-membered aromatic ring of the carbon skeleton of a ring being part of a condensed ring system formed by at least four rings, e.g. tetracycline
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/12—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having at least one of the hydroxy groups esterified by a carboxylic acid having the esterifying carboxyl group bound to a carbon atom of a ring other than a six-membered aromatic ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/02—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
    • C07C233/04—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals with carbon atoms of carboxamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C07C233/06—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals with carbon atoms of carboxamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a ring other than a six-membered aromatic ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C235/06—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2603/00—Systems containing at least three condensed rings
    • C07C2603/56—Ring systems containing bridged rings
    • C07C2603/58—Ring systems containing bridged rings containing three rings
    • C07C2603/70—Ring systems containing bridged rings containing three rings containing only six-membered rings
    • C07C2603/74—Adamantanes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Cosmetics (AREA)
CZ2020529A 2020-09-23 2020-09-23 Lipidoidy pro transfekci nukleových kyselin a jejich použití CZ2020529A3 (cs)

Priority Applications (13)

Application Number Priority Date Filing Date Title
CZ2020529A CZ2020529A3 (cs) 2020-09-23 2020-09-23 Lipidoidy pro transfekci nukleových kyselin a jejich použití
IL291097A IL291097B2 (en) 2020-09-23 2021-07-23 Lipidoids for nucleic acid transfection and their use
CN202180008677.8A CN114945556B (zh) 2020-09-23 2021-07-23 用于核酸转染的类脂质及其用途
BR112022024346A BR112022024346A2 (pt) 2020-09-23 2021-07-23 Lipidóides, agente e partícula de transfecção, e, uso do lipidóide ou do agente de transfecção ou da partícula de transfecção
JP2022530325A JP7260717B2 (ja) 2020-09-23 2021-07-23 核酸トランスフェクションのためのリピドイドおよびその使用
AU2021348717A AU2021348717B2 (en) 2020-09-23 2021-07-23 Lipidoids for nucleic acid transfection and use thereof
CA3154983A CA3154983C (en) 2020-09-23 2021-07-23 Lipidoids for nucleic acid transfection and use thereof
KR1020227011339A KR20220057605A (ko) 2020-09-23 2021-07-23 핵산 형질감염을 위한 리피도이드 및 그 용도
PCT/CZ2021/050079 WO2022063350A1 (en) 2020-09-23 2021-07-23 Lipidoids for nucleic acid transfection and use thereof
EP21762590.4A EP4031522A1 (en) 2020-09-23 2021-07-23 Lipidoids for nucleic acid transfection and use thereof
MX2022013522A MX2022013522A (es) 2020-09-23 2021-07-23 Lipidoides para la transfeccion de acidos nucleicos y su uso.
US18/015,254 US20230265049A1 (en) 2020-09-23 2021-07-23 Lipidoids for Nucleic Acid Transfection and Use Thereof
ZA2022/03990A ZA202203990B (en) 2020-09-23 2022-04-07 Lipidoids for nucleic acid transfection and use thereof

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CZ2020529A CZ2020529A3 (cs) 2020-09-23 2020-09-23 Lipidoidy pro transfekci nukleových kyselin a jejich použití

Publications (1)

Publication Number Publication Date
CZ2020529A3 true CZ2020529A3 (cs) 2022-03-30

Family

ID=77543261

Family Applications (1)

Application Number Title Priority Date Filing Date
CZ2020529A CZ2020529A3 (cs) 2020-09-23 2020-09-23 Lipidoidy pro transfekci nukleových kyselin a jejich použití

Country Status (13)

Country Link
US (1) US20230265049A1 (ko)
EP (1) EP4031522A1 (ko)
JP (1) JP7260717B2 (ko)
KR (1) KR20220057605A (ko)
CN (1) CN114945556B (ko)
AU (1) AU2021348717B2 (ko)
BR (1) BR112022024346A2 (ko)
CA (1) CA3154983C (ko)
CZ (1) CZ2020529A3 (ko)
IL (1) IL291097B2 (ko)
MX (1) MX2022013522A (ko)
WO (1) WO2022063350A1 (ko)
ZA (1) ZA202203990B (ko)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115403474B (zh) * 2021-05-28 2024-03-22 北京启辰生生物科技有限公司 脂质化合物及其在核酸递送中的应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102008032594A1 (de) * 2008-07-11 2010-01-14 Qiagen Gmbh Transfektionslösung
AU2014224205C1 (en) * 2013-03-08 2019-04-04 Novartis Ag Lipids and lipid compositions for the delivery of active agents
CA2953341C (en) * 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
ES2799409T3 (es) * 2014-08-07 2020-12-17 Takeda Pharmaceuticals Co Lípido catiónico
GB201604235D0 (en) * 2016-03-11 2016-04-27 Ucl Business Plc Lipids and complexes for the delivery of biologically-active material to cells
CN110167922B (zh) * 2016-11-08 2023-09-19 特拉维夫大学拉莫特有限公司 用于核酸递送的阳离子脂质及其制备

Also Published As

Publication number Publication date
MX2022013522A (es) 2022-11-16
IL291097A (en) 2022-05-01
IL291097B2 (en) 2024-01-01
ZA202203990B (en) 2022-12-21
AU2021348717B2 (en) 2023-04-06
AU2021348717A1 (en) 2022-05-19
CN114945556B (zh) 2024-08-27
KR20220057605A (ko) 2022-05-09
JP2022554024A (ja) 2022-12-27
JP7260717B2 (ja) 2023-04-18
US20230265049A1 (en) 2023-08-24
IL291097B1 (en) 2023-09-01
CN114945556A (zh) 2022-08-26
BR112022024346A2 (pt) 2023-04-04
CA3154983C (en) 2023-01-31
EP4031522A1 (en) 2022-07-27
CA3154983A1 (en) 2022-03-31
WO2022063350A1 (en) 2022-03-31

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